Overview
This is a Phase 2, single-center, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age. Up to 20 patients are planned to be enrolled. Patients will take a daily subcutaneous dose of open-label setmelanotide for up to 26 weeks.
Eligibility
Key Inclusion Criteria:
- Confirmed diagnosis of Prader-Willi Syndrome (PWS)
- Age 6 to 65
- BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th Percentile for age and sex
- Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
- Patient and/or guardian is able to communicate well with the Investigator, understand and comply with the requirements of the study, and understand English and sign the written informed consent.
Exclusion Criteria:
- Use of weight modulating medications
- Abnormal HbA1c, eGFR, ALT, AST, bilirubin, T4 values
- Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
- Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose
- Hypersensitivity to setmelanotide
- Diagnosis of severe psychiatric disorders
- Pregnant and/or breastfeeding
Other protocol defined Inclusion/Exclusion criteria may apply.